P589 关于奥达帕替尼治疗炎症性肠病的真实世界数据:安全高效,患者反馈极好

C Harris, T Gee, A Barcan, Y Yanagisawa, M Brown, J N Gordon
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Methods We prospectively collected data on all IBD patients treated with upadacitinib between November 2022 and November 2023 in a large NHS Trust serving approximately 1% of the population of England. The primary objective was to assess patient response to induction at 8 weeks (UC) and 12 weeks (CD). Demographic details, biochemical markers (faecal calprotectin and CRP) and disease activity scores were recorded. We also undertook a bespoke anonymised electronic survey to assess the patient experience and views on treatment with upadacitinib in comparison to previous treatments. Results Forty-two patients were included in the study (34 UC/8 CD). The average age was 41 (range 18-76) and 27 (64%) were male. 11/34 UC patients were biologic naïve. All CD patients were biologic experienced with the majority exposed to an anti-TNF, vedolizumab and ustekinumab. Overall, 34/40 (85%) patients responded to induction treatment based on disease activity scores (27 UC, 7CD), with 68% (22 UC, 5 CD) in remission. Data was missing for two patients. Response rates were similar between biologic naïve and biologic exposed patients (82% and 86% respectively). In the UC cohort, mean calprotectin at baseline improved from 1718ug/g (range 8-6000ug/g) to 311ug/g (4-3014ug/g). In the CD cohort, mean calprotectin improved from 1719ug/g (115-5874ug/g) to 314ug/g (4-917ug/g). 3/42 (7%) of patients discontinued upadacitinib due to disease progression with the remaining 93% continuing treatment. Our patient survey results revealed very high satisfaction with treatment (85%), with the vast majority preferring treatment with upadacitinib to their previous biological therapy. 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引用次数: 0

摘要

背景 乌帕他替尼是一种选择性 Janus 激酶抑制剂,最近在英国获准用于治疗溃疡性结肠炎(UC)和克罗恩病(CD),但缺乏实际数据。此外,与生物疗法相比,患者报告的小分子药物治疗经验方面的信息非常有限。本研究旨在提供有关达帕替尼治疗 IBD 疗效的真实世界数据,同时收集患者报告的有关达帕替尼治疗的可接受性和体验的具体反馈信息。方法 我们前瞻性地收集了2022年11月至2023年11月期间在一家大型NHS信托机构接受奥达帕替尼治疗的所有IBD患者的数据,该信托机构为英格兰约1%的人口提供服务。主要目的是评估患者在8周(UC)和12周(CD)时对诱导治疗的反应。我们记录了详细的人口统计学资料、生化指标(粪便钙蛋白和 CRP)和疾病活动评分。我们还进行了一项定制的匿名电子调查,以评估患者的治疗体验和对达达替尼治疗的看法,并与之前的治疗方法进行比较。结果 42 名患者参与了研究(34 名 UC/8 名 CD)。平均年龄为41岁(18-76岁不等),男性27人(64%)。11/34名UC患者是生物制剂新手。所有 CD 患者都有过生物制剂治疗经验,其中大多数都接触过抗 TNF、维多珠单抗和乌司替尼。总体而言,根据疾病活动评分,34/40(85%)名患者对诱导治疗做出了反应(27 名 UC 患者,7 名 CD 患者),68%(22 名 UC 患者,5 名 CD 患者)的病情得到缓解。有两名患者的数据缺失。未接受生物制剂治疗的患者和接受生物制剂治疗的患者的应答率相似(分别为82%和86%)。在 UC 组群中,基线时的平均钙蛋白从 1718ug/g(范围 8-6000ug/g)降至 311ug/g(4-3014ug/g)。在 CD 组中,平均钙蛋白从 1719ug/g (115-5874ug/g) 降至 314ug/g (4-917ug/g)。3/42(7%)的患者因疾病进展而中止了达达替尼治疗,其余 93% 的患者继续接受治疗。我们对患者的调查结果显示,他们对治疗的满意度非常高(85%),绝大多数患者更愿意接受达达替尼治疗,而不是之前的生物疗法。结论 在这项真实世界的研究中,奥达替尼诱导治疗的耐受性良好,在包括许多高度难治性疾病患者在内的混合患者群中显示出良好的疗效和极高的应答率和缓解率。没有出现意外的安全性信号。患者的体验非常积极。如果这一数据能在更大规模的研究中得到验证,那么在晚期疗法的排序中更早引入奥达帕替尼的理由就会越来越充分。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
P589 Real-world data on upadacitinib in the treatment of inflammatory bowel disease: safe and highly effective with extremely positive patient feedback
Background Upadacitinib is a selective Janus kinase inhibitor that has recently been approved in England for the treatment of ulcerative colitis (UC) and Crohn’s disease (CD) though real-world data is lacking. Furthermore, there is very limited information available on patient reported experiences of treatment with a small molecule compared with biological therapies. The object of this study was to provide real-world data on the efficacy of upadacitinib in the treatment of IBD in conjugation with collecting specific patient-reported feedback on acceptability and experience of treatment with upadacitinib. Methods We prospectively collected data on all IBD patients treated with upadacitinib between November 2022 and November 2023 in a large NHS Trust serving approximately 1% of the population of England. The primary objective was to assess patient response to induction at 8 weeks (UC) and 12 weeks (CD). Demographic details, biochemical markers (faecal calprotectin and CRP) and disease activity scores were recorded. We also undertook a bespoke anonymised electronic survey to assess the patient experience and views on treatment with upadacitinib in comparison to previous treatments. Results Forty-two patients were included in the study (34 UC/8 CD). The average age was 41 (range 18-76) and 27 (64%) were male. 11/34 UC patients were biologic naïve. All CD patients were biologic experienced with the majority exposed to an anti-TNF, vedolizumab and ustekinumab. Overall, 34/40 (85%) patients responded to induction treatment based on disease activity scores (27 UC, 7CD), with 68% (22 UC, 5 CD) in remission. Data was missing for two patients. Response rates were similar between biologic naïve and biologic exposed patients (82% and 86% respectively). In the UC cohort, mean calprotectin at baseline improved from 1718ug/g (range 8-6000ug/g) to 311ug/g (4-3014ug/g). In the CD cohort, mean calprotectin improved from 1719ug/g (115-5874ug/g) to 314ug/g (4-917ug/g). 3/42 (7%) of patients discontinued upadacitinib due to disease progression with the remaining 93% continuing treatment. Our patient survey results revealed very high satisfaction with treatment (85%), with the vast majority preferring treatment with upadacitinib to their previous biological therapy. Conclusion In this real-world study, induction therapy with upadacitinib was well tolerated and demonstrated good efficacy with excellent response and remission rates in a mixed patient cohort that included many with highly refractory disease. No unexpected safety signals were seen. The patient experience was overwhelmingly positive. If this data is replicated in larger studies there is an increasingly strong rationale for introducing upadacitinib earlier in the sequencing of advanced therapies.
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